Dundaru-Bandi Dominique, Niburski Kacper, Pitt Rebecca, Mohamed Nada, Gonzalez Cardenas Victor Hugo, Einhorn Lisa M, Ingelmo Pablo
Faculty of Medicine and Health Sciences, McGill University, Montreal, QC, Canada.
Department of Family Medicine, University of British Columbia, Vancouver, Canada.
Paediatr Drugs. 2025 May 10. doi: 10.1007/s40272-025-00698-2.
The evidence to support the efficacy and safety of pharmacological treatments for chronic non-cancer pain in children is limited. In practice, clinicians are often required to establish therapeutic plans using data extrapolated from adult studies, which may not apply to younger patients. Recent systematic reviews and meta-analyses indicate minimal evidence of benefit for these treatments in children; however, the low quality of studies included in these reviews complicates the conclusions that can be derived from them. In this article, we focus on safety, an outcome as critical as efficacy in clinical trial design but often designated as secondary or even exploratory. Specifically, we examine methods for assessing adverse events in clinical research and propose a practical approach for evaluating these events in everyday practice. Additionally, we outline our strategy to conduct a risk-benefit analysis at the individual patient level, highlighting the importance of using a composite risk-benefit metric rather than assessing these outcomes separately. This approach enables real-time monitoring of both drug-related symptom relief and adverse effects, facilitating clinically meaningful risk-benefit discussions with patients and their families. Finally, we advocate for improvements in clinical trial design for pediatric chronic pain treatments, particularly around adverse events. Future trials should incorporate standardized definitions, comprehensive risk-benefit evaluations, and transparent outcome reporting. Implementing these changes may enhance decision-making by balancing the safety and the effectiveness of pharmacological treatments for children and adolescents with chronic pain.
支持药物治疗儿童慢性非癌性疼痛有效性和安全性的证据有限。在实际操作中，临床医生常常需要依据从成人研究中推断出的数据来制定治疗方案，而这些数据可能并不适用于年龄较小的患者。近期的系统评价和荟萃分析表明，几乎没有证据显示这些治疗方法对儿童有益；然而，这些评价所纳入研究的低质量使得从中得出的结论变得复杂。在本文中，我们关注安全性，这一结果在临床试验设计中与有效性同样关键，但通常被指定为次要甚至是探索性的。具体而言，我们审视了临床研究中评估不良事件的方法，并提出了在日常实践中评估这些事件的实用方法。此外，我们概述了在个体患者层面进行风险效益分析的策略，强调使用综合风险效益指标而非分别评估这些结果的重要性。这种方法能够实时监测与药物相关的症状缓解情况和不良反应，便于与患者及其家属进行具有临床意义的风险效益讨论。最后，我们主张改进儿童慢性疼痛治疗的临床试验设计，尤其是围绕不良事件方面。未来的试验应纳入标准化定义、全面的风险效益评估以及透明的结果报告。实施这些变革可能通过平衡药物治疗对患有慢性疼痛的儿童和青少年的安全性和有效性来加强决策制定。
